A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
Condition(s) studied
Investigational drug(s) / intervention(s)
SRP-9003: Solution for single IV infusion
Glucocorticoid: Oral tablet (prophylactic)
Study summary
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
Eligibility
Primary outcome measure(s)
- Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers — Baseline, Day 60
Trial sites (10)
| Facility | City | Region | Status |
|---|---|---|---|
| University of California, San Diego-Altman Clinical and Translational Research Institute | La Jolla | California | |
| Nationwide Childrens Hospital | Columbus | Ohio | |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | |
| Children's Hospital of The King's Daughter | Norfolk | Virginia | |
| University Hospital Leuven (UZ Leuven) | Leuven | Vlaams Brabant | |
| NMRC Gent (UZ Gent) | Ghent | Belgium | |
| Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum | Essen | North Rhine-Westphalia | |
| Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico | Milan | Italy | |
| Hospital Sant Joan de Deu | Barcelona | Spain | |
| Newcastle University | Newcastle upon Tyne | United Kingdom |
More Sarepta Therapeutics, Inc. trials in the USA
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06246513 on ClinicalTrials.gov ↗ ← All trials in the USA